JONATHAN DEVIN
Posted: Monday, July 9, 2012 4:37 pm

St. Jude Director of Cancer Center Initiates Data Sharing

St. Jude Children’s Research Hospital recently began publishing data from the whole genome sequences of 600 pediatric cancers so that researchers at other institutions can study them.

At a time when cancer research is at its most exciting, the work to find new pediatric treatments is just beginning, according to Richard Gilbertson, MD, PhD, director of the Comprehensive Cancer Center at St. Jude Children’s Research Hospital.

“This data comes with a lot of responsibility,” Gilbertson said. “We decided that we’re going to release all the data for free. It’s going to go on the web, and anyone can look at it. There’s so much information there, why should we be the only ones looking at it?”

The data include not only the whole genome sequences for cancers, but also for the normal tissues, so that the two can be compared and variances identified. Similar to understanding the psychological background of a serial murderer, cancer is a killer best understood by its origin.

“If you take one cancer, it’s made up of multiple types of cancer, and one of the biggest determinants is where it came from in the first place,” Gilbertson said. “How a cancer behaves has a lot to do with where it was born.

“We’ve done a lot of lab models to show that if you take the mutations that you see in cancer and put it in a cell, it will make that kind of cancer. The advantage of that is that we can now use those models to do high throughput drug screening, which we’ve got here at St. Jude, and which many other institutions don’t have.”

Gilbertson has spent years studying medulloblastomas, pediatric brain tumors, and so far, he said, St. Jude has been able to identify two mutations that would not have been discovered had the entire human genome not been mapped out almost a decade ago.

Medulloblastomas can be carved into four subgroups, and St. Jude researchers have ascertained that some mutations don’t always come from structural problems within the DNA.

It’s a bright ray of hope considering that 40 percent of pediatric brain tumor patients die.

Gilbertson went to medical school in his native northern England, specializing in pediatrics, but also did a mini-research degree in pediatric brain tumors.

“As part of the project I had to meet some kids who were dying of cancer,” he said. “It was horrendous to see it happening. There are lots of treatment protocols, but they just didn’t work. I felt there must be something science could do to improve this.”

He went on to get a PhD in brain tumor science and in 1998 won an international award for brain tumor research. Soon after, he was courted by research facilities in the United States, including St. Jude.

In August, 2000, Gilbertson packed up his wife and three children and moved to Memphis. Here, Gilbertson directs the Comprehensive Cancer Center, an organization within St. Jude that, in 2007, received a special designation from the National Cancer Institute. There are about 60 NCI Cancer Centers around the country, and St. Jude has held that designation for more than 30 years.

St. Jude is the only solely pediatric institute designated as a Comprehensive Cancer Center, denoting its involvement in critical epidemiology and cancer prevention.

Despite the introduction of genetic research into the field, there are still plenty of needles left in haystacks. For one, research pools for cancer are relatively small. There are about 3,000 cases of pediatric brain tumors in the country each year, of which about 180 will come to St. Jude. In total, some 450 children become new patients at St. Jude each year.

Each of those cases can be broken down into even smaller subgroups.

Gilbertson said St. Jude has taken the lead in several research consortia, like the Pediatric Brain Tumor Consortium, and other facilities like MD Anderson Cancer Center to link similar cases into studies.

“The more we divide it up, the more difficult it is to do the trial, but the more we divide it up, the more chance we have of giving the right treatment to the right patient,” Gilbertson said. “It’s the right balance, I think.”

Because the cases of particular subgroups of cancer are low, pharmaceutical companies do not necessarily invest a lot of money in research for new drugs, but Gilbertson hopes that will become easier as the causes of certain cancers become clearer.

St. Jude established its own in-house pharmaceutical company, which is also working to start new trials.

“It’s hard to do the initial biology and the modeling in the lab,” Gilbertson said. “That’s hard, but then of course we can say we’ve got this particular subgroup already and now we’ve got a great therapy. Say there’s 20 kids in North America every year with (that subgroup). That’s where consortia come in.”

At the same time, St. Jude is digging a two-story basement for its new tower, which will house its new proton beam therapy, one of very few available in the United States.

Gilbertson, who initially had some anxiety about moving to the United States, said that working for a world leader in research has been such a joy that he will probably never return to England.

“I don’t treat kids anymore, but for somebody who comes from a background of treating kids, I’ve always loved medicine and I’ve always loved biology,” he said. “To put the two together is really brilliant.”